Identifying weaknesses in a specific type of childhood cancer
Official title: Nominating vulnerabilities in fusion oncoprotein-driven rhabdomyosarcoma
Funded at Duke University · NIH-11200239 · Data updated 2026-11-12
This study is looking at a tough type of cancer called rhabdomyosarcoma that affects kids and young adults, and it aims to find new ways to treat it by understanding the genetic changes caused by a specific protein that drives the disease.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | May 16, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $182,090 |
| NIH project number | 7R21CA273282-03 |
| Project ID | NIH-11200239 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children and young adults diagnosed with fusion-positive rhabdomyosarcoma.
✕Less likely to help
Patients with other types of rhabdomyosarcoma or those whose cancer is not driven by the PAX3-FOXO1 fusion protein may not benefit from this research.
What this research studies
This research investigates rhabdomyosarcoma (RMS), a cancer affecting children and young adults, particularly focusing on the high-risk form driven by the PAX3-FOXO1 fusion protein. The project aims to uncover genetic and epigenetic changes that could be targeted for therapy, using advanced DNA and RNA sequencing techniques. By developing experimental models that can manipulate the expression of the fusion protein, the researchers hope to identify potential therapeutic vulnerabilities that could lead to more effective treatments. This work addresses a critical gap in current treatment options for patients with fusion-positive RMS.
Why it matters
Potential benefit: If successful, this research could lead to new targeted therapies for children and young adults with high-risk rhabdomyosarcoma, improving their chances of long-term survival.
How similar studies have gone: While research on rhabdomyosarcoma has been ongoing, this specific approach targeting the PAX3-FOXO1 fusion protein represents a novel effort to identify actionable therapeutic targets.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Skapek, Stephen X — Duke University
- Study coordinator: Skapek, Stephen X
Related research
Other NIH-funded projects on the same topics.
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Targeting CLK2 to Fight Rhabdomyosarcoma
Aims to see if blocking CLK2 can weaken rhabdomyosarcoma cancer cells, potentially offering a new treatment for kids and young adults.
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Epitranscriptomic Crosstalk in Alveolar Rhabdomyosarcoma
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.